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2025 年前三个季度，79 款新药首次获 NMPA 批准上市，包括全球首个获批的 GCG/GLP-1 双受体激动减重药物玛仕度肽、全球首个高选择性外周 κ 阿片受体激动剂镇痛药安瑞克芬、国产首个 EZH2 抑制剂泽美妥司他片... Q4，又有哪些重磅新药会在国内获批上市呢？ 本文将根据 Insight 数据库「中国上市策略& 时长预测」模块，选出其中 10 款重磅新药（排名不分先后）进行分享，仅供读者参阅。 信达生物 匹康奇拜单抗 2024 年 9 月 26 日，信达生物匹康奇拜单抗注射液（研发代号：IBI112）的新药上市申请获 CDE 受理，用于治疗中重度斑块状银屑病。 此次报上市是基于一项在中国中重度斑块状银屑病受试者中开展的 III 期注册临床研究 CLEAR-1（NCT05645627）的积极结果。 该研究于 2024 年 5 月顺利达成主要研究终点和全部关键次要研究终点，研究显示匹康奇拜单抗组达成皮损清除（PASI 90、PASI 75、PASI 100、sPGA 0/1 和 sPGA 0）和生活质量改善（DLQI 0/1）的受试者比例均显著优于安慰剂组。研究治疗期间，匹康奇拜单抗整体安全性良好，未发现新的安全性信号。 匹康奇拜单抗是全球首个注册 III 期临床首要研究终点中第 16 周达到 PASI 90 的受试者比例突破 80% 的 IL-23p19 抗体药物，同时在同类生物药中具有最长的维持期给药间隔（每 12 周一次），有望为中国中重度斑块状银屑病患者带来皮损清除、生活质量改善和用药便利性提升等多方面的综合获益。 除了 CLEAR-1 以外，匹康奇拜单抗当前还开展了多项临床研究，包括： 在中重度银屑病患者中开展的随机撤药再治疗的 III 期临床； 在中重度银屑病患者中开展生物制剂治疗转匹康奇拜单抗治疗的 II 期临床； 在中重度活动性溃疡性结肠炎患者中开展的 II 期临床。 恒瑞医药 瑞拉芙普-α 2024 年 9 月 20 日，恒瑞 1 类新药瑞拉芙普-α 注射液（研发代号：SHR-1701）的上市申请获 CDE 受理，适应症为联合氟尿嘧啶类和铂类药物用于局部晚期不可切除、复发或转移性胃及胃食管结合部腺癌（G/GEJA）的一线治疗。 SHR-1701 是恒瑞医药自主研发的一种抗 PD-L1/TGF-βRII 双功能融合蛋白。在剂量递增、剂量扩展和临床扩展的 I 期研究中，SHR-1701 单药用于胃癌末线人群显示出良好的抗肿瘤活性。在此基础上，恒瑞开展了一项 SHR-1701 联合化疗用于晚期 G/GEJA 的 III 期临床研究。 在 2024 年 ESMO 大会上，恒瑞首次以口头报告的形式公布了此项 III 期临床的结果。数据显示，SHR-1701 所取得的疗效数据显著，尤其是在降低疾病进展和死亡风险层面（HR 值）： 在意向治疗人群（ITT）中的中位 OS 为 15.8 个月（vs 安慰剂组 11.2 个月），HR 为 0.66，降低 34% 的疾病进展或死亡风险； 在 PD-L1 CPS≥5 人群中，中位 OS 为 16.8 个月（vs 安慰剂组 10.4 个月） ，HR 为 0.53，降低 47% 的疾病进展或死亡风险。 截图来源：Insight 数据库 诺和诺德 帕西生长素 2024 年 9 月 5 日，诺和诺德帕西生长素在国内申报上市。根据公开信息和临床试验进展，Insight 数据库推断本次申报的适应症为用于治疗因生长激素分泌不足而导致生长缓慢的儿童患者。 帕西生长素（Somapacitan）是一种每周一次长效人生长激素蛋白衍生物。与现有的每日一次生长激素相比，帕西生长素可以将每年的治疗次数从 365 次减少至 52 次，大大提升患者治疗的便利性和依从性。 目前该药已在美国、欧盟、日本等海外国家和地区获批，用于治疗儿童和成人的生长激素缺乏症。在中国，诺和诺德开展了两项帕西生长素研究，其中治疗因生长激素分泌不足而导致生长缓慢的儿童患者的 III 期临床研究已经完成。 截图来源：Insight 数据库 值得一提的是，帕西生长素已经于今年 4 月率先落地海南博鳌乐城国际医疗旅游先行区，用于在生长激素缺乏症所致生长迟缓的 3 岁及以上儿童和青少年中替代内源性生长激素，并在 5 月 24 日开出中国首张处方。 和美药业 莫米司特-Hemay005 2024 年 4 月 24 日，和美药业 1 类新药莫米司特-Hemay005 上市申请获 CDE 受理，用于中至重度斑块状银屑病。 在 2023 年美国风湿病学会年会（ACR）上，莫米司特治疗中至重度慢性斑块状银屑病的 III 期临床（NCT04839328）结果已公布。结果显示，莫米司特和安慰剂组 PASI75 比例分别为 53.6% vs 16.0%，sPGA 0/1 率分别为 31.3% vs 6.4%。 Insight 数据库显示，目前国内共有 2 款 PDE4 抑制剂获批上市，分别是阿普米司特（印度太阳制药/安进）和克立硼罗乳膏（辉瑞）。大冢制药的地法米司特、和美药业的莫米司特-Hemay005 均处于上市审评阶段，后者是首个国产 PDE4 抑制剂。 截图来源：Insight 数据库 此外，国内方面，恒瑞、中国生物制药、华东医药、济川药业、健康元等企业均在布局 PDE4 赛道，总共有 5 款 PDE4 抑制剂已进入 III 期临床。 GSK 玛贝兰妥单抗 2024 年 12 月 7 日，GSK 的 BCMA ADC 药物玛贝兰妥单抗在中国申报上市，玛贝兰妥单抗联合硼替佐米加地塞米松（BVd）治疗复发或难治性多发性骨髓瘤。 该申请基于 III 期头对头 DREAMM-7 试验的中期结果。数据显示，该试验达到了其主要终点，显示与达雷妥尤单抗联合硼替佐米和地塞米松（DVd）治疗复发或难治性多发性骨髓瘤相比，BVd 方案在 PFS 方面具有统计学显著和临床意义的改善。 在后续计划的中期分析中，DREAMM-7 试验达到了 OS 这一关键次要终点，表明与标准治疗 DVd 方案相比，BVd 方案显著降低了死亡风险。 CytokineticsAficamten 2024 年 10 月 18 日，箕星药业宣布 CDE 已正式受理艾非凯泰（Aficamten）用于治疗症状性梗阻性肥厚型心肌病（oHCM）的新药上市申请。 Aficamten 是一款在研的新一代选择性小分子心肌肌球蛋白抑制剂，其治疗 oHCM 获 FDA 和 NMPA 授予的突破性治疗药物认定。 2020 年 7 月，箕星与 Cytokinetics 就该药在大中华区的研发和商业化签订了独家许可协议。2024 年 12 月，箕星药业与赛诺菲共同宣布签订正式协议，协议约定赛诺菲将收购箕星在大中华区独家开发和商业化 Aficamten 的权益。 该新药上市申请是基于关键性全球 III 期临床研究 SEQUOIA-HCM 所取得的积极结果。研究结果表明，与安慰剂相比，Aficamten 治疗能显著提高患者的运动能力，治疗效果在所有预设亚组中都是一致的，这些亚组反映了患者的基线特征和治疗策略，包括接受或未接受作为背景治疗的 β-受体阻滞剂的患者。在所有 10 个预先设定的次要终点中，也观察到具有统计学意义和临床意义的改善。 辉瑞 马塔西单抗 2024 年 8 月 13 日，马塔西单抗（Marstacimab、PF-6741086）在国内报上市，这是辉瑞研发的一种血友病新型疗法，是一种靶向组织因子途径抑制物（TFPI）K2 结构域的单克隆抗体，属于血友病再平衡疗法。目前马塔西单抗已在美国和欧盟获批用于治疗血友病 A 及血友病 B。 马塔西单抗的获批是基于 III 期 BASIS 研究。该研究不伴抑制物队列的中期分析结果显示，在不伴抑制物的血友病 A 及血友病 B 受试者中，经过马塔西单抗 12 个月的积极治疗期后，与既往因子预防（RP）和按需治疗 (OD) 相比，其经治疗出血的年化出血率 (ABR) 分别降低了 35% 和 92%。 2025 年 2 月，马塔西单抗正式落地海南博鳌乐城先行区，通过预充式注射笔皮下注射固定剂量给药，只需每周一次，有望减少血友病患者因频繁输注带来的心理负担和用药负担。 精准生物 普基仑赛 2024 年 7 月 20 日，重庆精准生物自主研发的国家一类生物新药 pCAR-19B 细胞自体回输制剂（普基仑赛注射液）上市申请获 NMPA 正式受理，该产品是国内首款针对儿童白血病的 CAR-T 产品，用于治疗 3-21 岁患有 CD19 阳性复发/难治性急性淋巴细胞白血病（ALL）的患者。 在 2024 年 ASH 大会上，该药用于 CD19 阳性 R/R B-ALL 的 II 期临床结果首次被披露。中位随访 211 天，最佳 ORR 为 90.63%，其中 78.13% 的患者达到 CR，12.5% 的患者达到 CRi，超过了当前商业化抗 CD19 CAR-T 疗法产品在 R/R B-ALL 中报道的疗效。 此外，98.27% 的 ORR 患者获得了阴性微小残留病（MRD）阴性缓解。3 个月 ORR 为 76.56%。中位缓解持续时间（DOR）为 10.61 个月，中位总生存期（OS）为 23.92 个月。 根据 Insight 数据库，目前国内已有 4 款靶向 CD19 的 CAR-T 产品获批上市，分别为恒润达生的雷尼基奥仑赛、复星凯瑞引进的的阿基仑赛、药明巨诺的瑞基奥仑赛、合源生物的纳基奥仑赛。普基仑赛（精准生物）、IM19（艺妙神州/先声）、布瑞基奥仑赛（吉利德/复星凯瑞）均处于上市审评阶段。 截图来源：Insight 数据库 Alnylam/赛诺菲 芬妥司兰钠 2024 年 5 月 7 日，siRNA 疗法芬妥司兰钠上市申请获 CDE 受理，拟用于作为常规预防治疗，用于有或无凝血因子 VIII 或 IX 抑制物的血友病 A 或 B 的成人患者和 ≥12 岁青少年患者，以预防或减少出血的发生频率。 芬妥司兰钠来自于 Alnylam，2018 年 8 月，赛诺菲和 Alnylam 公司达成合作，获得 Fitusiran 的全球开发和商业化权利。该药采用 Alnylam Pharmaceutical 的 ESC-GalNAc 偶联技术，因此在皮下给药时具有更强的效力和持久性。 2025 年 3 月 28 日，该药在美国率先获批，商品名为 Qfitlia，成为美国首个获批的治疗 A 型或 B 型血友病（无论是否含有抑制剂）的疗法，每年只需注射六次即可提供持续保护，代表着血友病治疗的重要突破。 在中国，该产品还被 CDE 纳入了《以患者为中心的罕见疾病药物研发试点工作计划（「关爱计划」）》试点项目，这是一项旨在罕见疾病药物研发领域落实以患者为中心的药物研发理念的试点工作。 Ascendis/维昇药业隆培促生长素 2024 年 3 月 8 日，维昇药业宣布 NMPA 已受理隆培促生长素的上市许可申请。这是首个在美国及欧洲获批上市的长效生长激素（每周注射一次），用于治疗儿童生长激素缺乏症（PGHD）。 隆培促生长素由 Ascendis Pharma A/S 所有，并在全球范围内开发。2018 年 11 月，维昇药业从 Ascendis 引进了该产品在中国的开发和商业化权益。 2022 年 4 月，维昇药业在中国完成了隆培促生长素治疗 PGHD 的关键 III 期临床试验。 结果显示，隆培促生长素 52 周年化生长速率（AHV）高于短效（每日注射）人生长激素，具有统计学显著差异，并可能使患有 PGHD 的儿童在有限的时间窗口内更有效达到治疗目标。第 52 周时，隆培促生长素 AHV 为 10.66 厘米/年，而短效（每日注射）人生长激素为 9.75 厘米/年（p=0.001）。 如欲了解更多国内新药获批情况，可以扫描文末二维码，前往 Insight 数据库查询。 封面来源：站酷海洛题图 免责声明：本文仅作信息分享，不代表 Insight 立场和观点，也不作治疗方案推荐和介绍。如有需求，请咨询和联系正规医疗机构。 编辑：ccai PR 稿对接：微信 insightxb 投稿：微信 insightxb；邮箱 insight@dxy.cn

iStock, Grandbrothers From more than 30 target action dates in the last three months of the year, BioSpace has narrowed the list to six regulatory decisions that could have far-reaching implications for biopharma and patients. As 2025 enters its home stretch, the FDA is also nearing the end of its calendar. But that doesn’t mean that the industry is in for a quiet year-end—quite the opposite, in fact. In the last three months of 2025, the FDA is set to decide on at least 30 regulatory applications, many of which could help shift the treatment landscape for hard-to-treat diseases or push the boundaries for novel modalities. One upcoming verdict, for instance, could mark another industry first for CAR T therapies, while another could mean the comeback of a previously-pulled cancer drug. Here, BioSpace rounds up six of these most highly anticipated decisions in Q4, looking at the data backing their bids and what their approvals could mean for the industry. After Delay, Regeneron Awaits Two Eylea HD Verdicts Regeneron is looking to boost the pro high-dose Eylea with a move into macular edema after retinal vein occlusion (RVO) and a monthly dosing regimen across all its approved indications. The FDA is currently reviewing these applications and is expected to release its verdict in the fourth quarter. No specific decision date has yet been indicated. The regulator had an initial target action date of Aug. 19, but extended the review period due to issues at a third-party manufacturing site, Regeneron announced earlier that month. The production plant in contention was previously owned by the CDMO giant Catalent, which was acquired by Novo Nordisk’s parent company for $16.5 billion in February 2024 . An FDA report made public in August revealed uncontrolled and unaddressed contaminations at this site, including with bacteria and cat hair. Regeneron has nevertheless reassured investors that aside from these third-party problems, the FDA has not flagged any problems with the submission. “Based on our discussions, we believe that there’s nothing significant left to be done,” CEO Leonard Schleifer said during the company’s Q2 earnings call . “We are expecting, once the resolution of the filling issues has occurred, to receive favorable action, we hope, from the FDA.” To support its application for high-dose Eylea, Regeneron filed data from the Phase III QUASAR study, which demonstrated that the high-dose formulation was non-inferior to the currently approved standard-dose version of Eylea when used to treat patients with macular edema after RVO, according to a readout in December 2024. Adverse events were comparable between the two formulations. High-dose Eylea is currently approved for wet age-related macular degeneration, diabetic macular edema and diabetic retinopathy. GSK’s Blenrep Comeback Bid Nears Moment of Truth By Oct. 23, the FDA is expected to release its decision on GSK’s proposal to bring Blenrep back to the market, this time as a second-line treatment for relapsed/refractory multiple myeloma. Blenrep, an antibody-drug conjugate targeting the CD38 protein, was originally approved in August 2020 under the FDA’s accelerated pathway. But soon after failing its confirmatory Phase III study in November 2022, GSK withdrew the product from the market. Then, last year, GSK started setting the stage for Blenrep’s comeback with data from the Phase III DREAMM-7 trial. A readout in February 2024 demonstrated a significant 59% drop in the risk of death or disease progression in patients treated with Blenrep plus bortezomib and dexamethasone, as compared with a similar regimen using Johnson & Johnson’s Darzalex. Then, in June that same year, the pharma reported findings from another late-stage study, DREAMM-8, touting significantly better progression-free survival for Blenrep versus Takeda’s Velcade. GSK is using these two studies to support its regulatory application for Blenrep, but in July this year, the FDA’s internal reviewers flagged “high rates of ocular toxicity and dose modifications” in both trials. The DREAMM studies also had “limited dose exploration” which in turn highlight the unaddressed need for a “careful evaluation” of Blenrep’s risks in its proposed indications, according to the reviewers. An external advisory committee in July agreed with this assessment and voted 7–1 against Blenrep , finding an unfavorable benefit-risk pro the drug in its proposed indication. “The efficacy data were strong but the toxicity data were also very strong,” panelist Neil Vasan said at the time. Shortly after the vote, the FDA extended Blenrep’s review by three months. BMS’ Breyanzi Could Become First CAR T Therapy for Marginal Zone Lymphoma Bristol Myers Squibb is proposing its CAR T therapy Breyanzi for the treatment of marginal zone lymphoma (MZL), potentially blazing the trail for the therapeutic class in this indication. The FDA has a target decision date of Dec. 5. To support its application, BMS filed findings from the Phase II TRANSCEND FL study, a single-arm, open-label trial that enrolled more than 270 patients with relapsed or refractory follicular lymphoma or MZL. Data presented at the 2025 International Conference on Malignant Lymphoma in June demonstrated a 95.5% overall response rate in patients with MZL, including 62.1% who saw a complete response. The safety pro TRANSCEND FL was consistent with what had previously been established for Breyanzi. Rates of cytokine release syndrome and neurologic complications were “low,” according to BMS, with no grade 4 or 5 cases detected. If it wins the FDA’s blessing, Breyanzi would be the first CAR T therapy indicated for MZL, according to an August 2025 news release from the company. It would also mark the second time that Breyanzi notched a regulatory record: In March 2024, it became the first CAR T to win approval for chronic lymphocytic leukemia and small lymphocytic leukemia in relapsed/refractory patients. Breyanzi works by binding to the CD19 protein, in turn triggering the activation and action of CAR-T cells, as well as the release of pro-inflammatory signals to destroy the target tumor. It is also approved for large B-cell lymphoma, follicular lymphoma and mantle cell lymphoma. The drug’s label carries a boxed warning for cytokine release syndrome, neurological toxicities and secondary hematological malignancies. Arrowhead Makes Case for RNAi Therapy in Rare Cholesterol Disease After delivering four firsts for the rare disease space in August, the FDA by Nov. 18 could grant another—this one for Arrowhead Pharmaceuticals’ plozasiran , an RNA interference therapeutic for familial chylomicronemia syndrome (FCS). Afflicting 1 to 2 people per million , FCS is an ultra-rare, heritable disease that manifests as excessively high levels of triglyceride levels, typically reaching over 880 mg/dL, according to Arrowhead . In turn, patients with FCS suffer from cardiometabolic and other complications such as diabetes, hepatic steatosis, abdominal pain and even cognitive problems. There are currently no treatments that can adequately control FCS in the U.S., according to Arrowhead. Arrowhead’s answer to FCS is plozasiran , an investigational therapy that reduces the production of the ApoC3 protein, which is naturally produced in liver cells and serves as a component of triglyceride-rich lipoproteins. ApoC3 prevents the breakdown and clearance of these fatty molecules. By reducing ApoC3 production, plozasiran helps normalize lipid levels in FCS patients. Arrowhead is supporting its application with data from the Phase III PALISADE trial. A readout in November 2024 demonstrated that 25 mg of plozasiran—the proposed commercial dose—led to a roughly 80% reduction in triglyceride levels. In at least half of the treated patients, triglyceride levels stayed below 500 mg/dl, a threshold linked with worse acute pancreatitis risk. Plozasiran treatment also resulted in lower cholesterol levels. Arrowhead is also developing plozasiran for severe hypertriglyceridemia and dyslipidemia. Ascendis Eyes Approval in Rare Bone Growth Disorder Also awaiting a rare disease decision is Ascendis Pharma, which is advancing TransCon CNP, also known as navepegritide, for the treatment of children with achondroplasia. The FDA is set to release its verdict by Nov. 30. Achondroplasia is a rare, genetic disease characterized by dwarfism due to faulty bone development. Ascendis is targeting a specific disease type caused by mutations in the FGFR3 gene, resulting in manifestations in different tissues across the body. Aside from bone problems, patients with this specific type of achondroplasia also suffer from muscular, neurological and cardiorespiratory problems. Around 250,000 people worldwide are affected by this disease, according to Ascendis. TransCon CNP works by providing sustained exposure to the C-type natriuretic peptide, a molecule that helps stimulate bone growth and counter the inhibiting effects of FGFR3 mutations. Through this mechanism, Ascendis contends that its drug candidate could help ease the burden of symptoms and complications in achondroplasia. Ascendis tested the TransCon CNP in ACcomplisH, a randomized, double-blinded and placebo-controlled Phase II study that enrolled 42 patients who received 6 μg, 20 μg or 50 μg of the drug. Results, published in The Lancet in November 2023, showed that patients on TransCon CNP saw significant improvements in annualized growth velocity, alongside other key benefits, such as a stabilized quality of life and better physical functioning. In a June 2025 statement , Ascendis Chief Medical Officer Aimee Shu said the biotech’s clinical development program for TransCon CNP is the “first ever to demonstrate improvements beyond linear growth at 52 weeks compared to placebo.” Cytokinetics Could Challenge BMS in Cardiomyopathy Market Closing out this list is Cytokinetics and its cardio candidate aficamten, which is currently under FDA review for obstructive hypertrophic cardiomyopathy (oHCM). The FDA’s target decision date is Dec. 26. Aficamten blocks the cardiac myosin protein, which plays a role in heart contraction, allowing the oral drug candidate to address a key symptom of oHCM. Data from the Phase III MAPLE-HCM study, presented in August at the 2025 European Society of Cardiology Congress, demonstrated a significant improvement in exercise capacity over metoprolol, a beta-blocker used as standard-of-care in the disease. At the time, analysts at Truist Securities said aficamten’s results position it to be “potentially best-in-class” in oHCM. They called the MAPLE-HCM data “robust,” noting that outperforming metoprolol could help aficamten distinguish itself in the oHCM market. Cytokinetics filed for aficamten’s approval in late 2024, at which time the FDA provided an initial target action date of Sept. 26, 2025 . In May, however, the regulator extended its review by three months to review an additional risk mitigation strategy submission. If approved, aficamten will compete with BMS’s Camzyos, also a cardiac myosin inhibitor that won approval for oHCM in 2022 . The pharma is testing the drug in a non-obstructive form of the heart disease, but a readout in April this year was disappointing : Camzyos was unable to significantly outperform placebo in terms of exercise capacity outcomes, nor did it lead to better physical function.